A Clinical Study of ANX005 and IVIG in Subjects With Guillain Barré Syndrome (GBS)

Phase 1

Completed

• Conditions: Guillain-Barré Syndrome
• Interventions: Drug: ANX005; Drug: Intravenous immunoglobulin

• Registration Number: NCT04035135
• Lead Sponsor: Annexon, Inc.

Brief Summary

This study is a multi center, open-label, study of ANX005 in combination with IVIg in subjects diagnosed with GBS.

Detailed Description

The study will enroll approximately 12 subjects into one cohort, with subjects receiving 75 mg/kg of ANX005, a dose shown to be safe and well-tolerated as monotherapy and that provides a meaningful level of exposure to be tested in combination with IVIg. Dosing of IVIg will be administered as is the current standard of care (0.4 g/kg/day x 5 days) and ANX005 will be administered concurrently, beginning on Day 1 or 2 of the treatment period. Subjects will be followed for 6 months after treatment for observation and evaluation.

Study Timeline

• Study First Submitted: 2019-07-11
• Study First Submitted QC: 2019-07-26
• Study First Posted: 2019-07-29
• Study Start: 2020-01-29
• Primary Completion: 2021-05-19
• Study Completion: 2021-05-19
• Status Verified: 2021-08
• Last Update Submitted: 2021-08-09
• Last Update Posted: 2021-08-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 14

Inclusion Criteria

• Diagnosis of GBS according to the National Institute of Neurological Disorders and Stroke Diagnostic Criteria for Guillain Barré Syndrome
• Onset of GBS-related weakness ≤14 days prior to infusion
• GBS-DS score of 3, 4, or 5

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Exclusion Criteria

• Clinically significant findings that may interfere with the conduct of the study or the interpretation of the data
• Be at risk of suicide or self-harm
• Received previous treatment with plasma exchange for GBS
• Any diagnosis of a variant of GBS
• Have a history of anaphylaxis or severe systemic response to immunoglobulin
• Documented, clinically significant, pre-existing polyneuropathy from another cause
• Clinically significant intercurrent illness, medical condition, or medical history
• History of chronic use of steroid or immunosuppressant medication
• Active alcohol, drug, or substance abuse
• Females who are pregnant, breast feeding, or unable or unwilling to use highly effective methods of contraception throughout the study

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Open Label Treatment Arm	ANX005	One (1) dose of ANX005, 75 mg/kg, will be administered IV. IVIg, 0.4 g/kg, will be administered for 5 consecutive Days.
Open Label Treatment Arm	Intravenous immunoglobulin	One (1) dose of ANX005, 75 mg/kg, will be administered IV. IVIg, 0.4 g/kg, will be administered for 5 consecutive Days.

Primary Outcome Measures

Name	Time	Method
Safety and tolerability of ANX005 when administered in combination with IVIg: incidence of TEAEs, SAEs, AE's	6 months	As measured by incidence of TEAEs, SAEs, AE's related to ANX005, SAE's related to ANX005, Grade 3 or higher AEs, Grade 3 or higher AEs related to ANX005, AEs leading to study or treatment discontinuation.

Secondary Outcome Measures

Name	Time	Method
Values and change from baseline in Guillain-Barré Syndrome Disability Score (GBS-DS) by visit	6 months	The 6-point Guillain-Barré Syndrme Disability Score (GBS-DS) is a widely accepted and easily obtainable scoring system used to assess functional status of GBS subjects. The score is as follows: 0 = Healthy, 1 = Minor symptoms and capable of running, 2 = Able to walk independently 10 meters or more but unable to run, 3=Able to walk more than 10 meters across an open space with help, 4 = Bedridden or chair bound, 5 = Needing mechanical ventilation, 6 = death
Pharmacodynamics of ANX005 when administered in combination with IVIg	4 months	As measured by CH50 and C1q serum concentrations
Pharmacokinetics of ANX005 when administered in combination with IVIg	3 months	As measured by ANX005 serum concentrations

Trial Locations

Locations (2)

National Institute of Neurosciences and Hospital; 🇧🇩 Dhaka, Bangladesh

Aarhus University Hospital; 🇩🇰 Aarhus, Denmark